Hey mate, I appreciate the reply.
I had a quick look online for some research into the expected decrease in PUL 2.0 scores for a non ambulant DMD population, and found the following study: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6010252/
The study tested a population of both ambulant and non ambulant boys (total pop = 187) and measured their PUL 2.0 scores over 24 months. The results found decreases for both ambulant (mean = -2.07) and non-ambulant (mean = -4.36) for PUL 2.0+ scores over this period. The regression used for the data for both were linear, so let’s presume the average decrease for the relevant non-ambulant population for comparison is -4.36/4 for 6 months of the study, yielding a -1.09 average PUL 2.0 decrease. I would think this is still a significant distance from the conservative +0.125 value we get to from the P2A results (removing patient 8’s +7.0 score).
I’ll have to look into the lymphocyte data in the P2A trial results to really comment, but I think we can only hope that the 25mg and 50mg stratums yield good results in P2B (for both us as shareholders and for these poor boys).
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